PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli
The EMA's human medicines advisory committee has recommended that BioMarin Pharmaceutical's haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to
In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.
BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PK
US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemop
Endeavor BioMedicines has completed a third-round financing, raising an impressive $132.5 million to fund trials of its two clinical-stage drug candidates for pulmonary fi
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.